Eficácia da midostaurina na mastocitose sistêmica – um relato de caso
Efficacy of midostaurin in systemic mastocytosis: a case report
Stéphanie Kim Azevedo de Almeida; Igor Rafael Guedes Pereira Brandão; Marina França de Paula Santos; Jorge Kalil; Pedro Giavina Bianchi
Resumo
Paciente do sexo feminino, com 63 anos de idade, portadora de mastocitose sistêmica há cerca de 20 anos, sendo agressiva há 10 anos. Crises quase diárias com manifestações do trato gastrointestinal e vasomotoras. Após diversas tentativas de tratamento, iniciou uso de midostaurina, um inibidor multiquinase. Depois de 6 meses de uso, observou-se bom controle dos sintomas, diminuição em quase 50% da triptase sérica e desaparecimento completo das lesões cutâneas.
Palavras-chave
Abstract
A 63-year-old female presented with an approximately 20-year history of systemic mastocytosis, which had become aggressive over the past 10 years. She experienced almost daily episodes of gastrointestinal and vasomotor manifestations. After multiple treatment attempts, she was started on midostaurin, a multikinase inhibitor. At 6 months of therapy, satisfactory control of symptoms was achieved, with a nearly 50% reduction in serum tryptase and complete resolution of cutaneous lesions.
Keywords
References
1. Pardanani A. Systemic mastocytosis in adults: 2021 Update on diagnosis, risk stratification and management. Am J Hematol. 2021;96(4):508-25.
2. Burgard C, Rosar F, Khreish F, Ezziddin S. Systemic Mastocytosis Treatment with Midostaurin: [18F]FDG PET/CT as a Potential Monitoring Tool for Therapy Outcome. Diagnostics (Basel). 2022;12(3):680.
3. Miettinen M, Lasota J. KIT (CD117): a review on expression in normal and neoplastic tissues, and mutations and their clinicopathologic correlation. Appl Immunohistochem Mol Morphol. 2005;13(3):205-20.
4. Velloso EDRP, Padulla GA, de Cerqueira AMM, de Sousa AM, Sandes AF, Traina F, et al. Diagnosis and treatment of systemic mastocytosis in Brazil: Recommendations of a multidisciplinary expert panel. Hematol Transfus Cell Ther. 2022;44(4):582-94.
5. Lim KH, Tefferi A, Lasho TL, Finke C, Patnaik M, Butterfield JH, et al. Systemic mastocytosis in 342 consecutive adults: survival studies and prognostic factors. Blood. 2009;113(23):5727-36.
6. Gotlib J, Kluin-Nelemans HC, George TI, Akin C, Sotlar K, Hermine O, et al. Efficacy and Safety of Midostaurin in Advanced Systemic Mastocytosis. N Engl J Med. 2016;374(26):2530-41.
7. Krauth MT, Mirkina I, Herrmann H, Baumgartner C, Kneidinger M, Valent P. Midostaurin (PKC412) inhibits immunoglobulin E-dependent activation and mediator release in human blood basophils and mast cells. Clin Exp Allergy. 2009;39(11):1711-20.
8. Perez IL, Giavina-Bianchi M, Mamede LQ, Antila HG, Pereira GF, Kalil J, et al. Escabiose mascarada por mastocitose sistêmica. Arq Asma Alerg Imunol. 2020;4(1):141-4.
9. Chandesris MO, Damaj G, Canioni D, Brouzes C, Lhermitte L, Hanssens K, et al.; CEREMAST Study Group. Midostaurin in Advanced Systemic Mastocytosis. N Engl J Med. 2016;374(26):2605-7.
10. Rossignol J, Nizard S, Blanc AS, Filipovics A, Lortet-Tieulent J, Bouktit H, et al. Therapeutic management and outcome of patients with advanced systemic mastocytosis treated with midostaurin: A comprehensive real-life study in the French national healthcare database. Hematol Oncol. 2022;40(5):1030-40.
11. Gotlib J, Kluin-Nelemans HC, George TI, Akin C, Sotlar K, Hermine O, et al. Efficacy and Safety of Midostaurin in Advanced Systemic Mastocytosis. N Engl J Med. 2016;374(26):2530-41.
Submitted date:
09/12/2023
Accepted date:
11/12/2023
Facebook
Google+
Twitter
LinkedIn
Mendeley
StumbleUpon
CiteULike
Reddit
Email